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VEGF inhibitor PTC299

Phase 1

Brain and Central Nervous System Tumors | Small molecule | Oncology |PTC Therapeutics, Inc.|Last Updated: May 4, 2015

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment28
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System TumorsPHASE1 COMPLETED 28 — — Nov 1, 2010Jan 1, 2015May 4, 20158 United States
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Study Endpoints
Primary Endpoints
Maximum-tolerated dose
First four weeks of treatment
Adverse events
From the first day of treatment until 30 days after the last dose
Secondary Endpoints
Percentage of study participants with complete response or partial response to the study treatment
Every 8 weeks
Pharmacokinetics
Day 1 and day 28 of course 1
Change from baseline in blood angiogenic markers and cytokines at discontinuation or completion of treatment
Before the first dose of drug on day 1 of course 1 and at the discontinuation or completion of treatment
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Interventions
NameTypeDescription
VEGF inhibitor PTC299DRUGThis is a dose escalation study. Study participants will receive .6 or 1.2 mg/kg orally twice daily or 1.2, 1.5, or 2.0 mg/kg orally three times daily for four consecutive weeks (a course). In the absence of unacceptable toxicity or disease progression, treatment may continue for up to 12 courses (approximately one year)
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Eligibility Criteria
Age Range3 Years — 21 Years
SexALL
Healthy VolunteersNo
Study Sites8

DISEASE CHARACTERISTICS: * Histologically confirmed diagnosis of primary central nervous system (CNS) malignancy at time of diagnosis or recurrence * Histology verification not required for intrinsic brain stem tumors and optic pathway gliomas * Must have radiographic evidence of progressio...

Countries:United States
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